BOTOX(RegisteredTM) Treatment in Pediatric Upper Limb Spasticity: Open-label Study. Protocol: 191622-105

Study ID
STU 112012-071

Cancer Related
No

Healthy Volunteers
No

Study Sites

Contact
Kara Lorduy
214-456-7000
kara.lorduy@childrens.com

Principal Investigator
Fatma Gul

Summary

This study will consist of approximately 12 clinic visits and up to 5 telephone follow-up calls over a period of approximately 60 weeks. at each treatment visit, the patient will receive at least 6 u/Kg and no more than 8 u/Kg of BoToX in the upper limb alone, or no more than 10 u/Kg of BoToX in both the upper and lower limbs combined. also, 5 telephone follow-ups will be made 2 weeks after each (re)treatment visit.

over the course of the study, each participant will complete the following study events:

* Screening visit (up to 4 weeks before day 1)
* 1st Treatment visit (day 1)
* Retreatment visits (up to 4 visits, at least 12 weeks since the last treatment visit)
* Telephone follow-up (5 calls, 2 weeks after each treatment)
* Follow-up visits (every 6 weeks post-treatment until retreatment)
* exit visit (12 weeks after the last treatment)

Participant Eligibility

The following are inclusion criteria for patients who are transitioning from Allergan Study 191622-101 (
* rollover
* patients):
1. Eligible patients who successfully completed Allergan Study 191622-101 without major protocol deviations (eg, noncompliance to protocol-required procedures) and who, in the investigator[Single Quote]s clinical judgment, did not experience an adverse event that may indicate an unacceptable safety risk for additional BOTOX treatments
2. Stable medical condition in the investigator[Single Quote]s opinion
3. Written informed consent has been obtained from parent/legally authorized representative
4. Written minor assent has been obtained in accordance with local laws and institutional review board (IRB)/independent ethics committee (IEC) requirements
5. Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable (eg, Written Authorization for Use and Release of Health and Research Study Information for US sites and written Data
Protection consent for European Union [EU] sites)
6. Negative urine pregnancy test at day 1 visit (for females of childbearing potential, defined as females post menarche)
7. Patients who are appropriate candidates for the protocol-specified study treatment doses per the investigator[Single Quote]s clinical judgment

The following are inclusion criteria for de novo patients:
1. Male or female, 2 to 16 years and 11 months of age (prior to 17th birthday) at the day 1 visit
2. Minimum weight of 10 kg at the screening and day 1 visits
3. Upper limb monoplegic, hemiplegic, or triplegic spasticity (spasticity confirmed by Hypertonia Assessment Tool [HAT]) with single-arm sparing (only 1 arm requiring botulinum toxin treatment for spasticity during the study) resulting from cerebral palsy, or post-stroke with onset prior to age 2 and at least 12 months prior to the day 1 visit
4. Patient[Single Quote]s spasticity meets at least one of the conditions below at the screening and day 1 visits:
a. Elbow flexor tone of 2 or greater as measured by the MAS-B AND elbow flexor muscle contracture of no more than 30 degrees, and/or
b. Wrist flexor tone of 2 or greater as measured by the MAS-B AND at least neutral position for wrist with fingers at maximum extension
5. Has demonstrable movement and attempted utilization of the study upper limb
6. Gross Motor Function Classification System x Expanded and Revised (GMFCS-E&R) level I to IV at the screening visit
7. Manual Ability Classification System (MACS) level I to IV at the screening visit
8. Stable medical condition in the investigator[Single Quote]s opinion
9. Acceptable clinical laboratory results at the screening visit
10. Patients who are on anti-spastic medications or muscle relaxants (eg, oral baclofen, tizanidine, dantrolene, scopolamine [oral or patch], vigabatrin, or benzodiazepine therapy) must be on a stable dose and regimen for at least 30 days prior to the day 1 visit
11. Patients who are on anti-epileptic medications must be on a stable dose and regimen for at least 30 days prior to the day 1 visit
12. Written informed consent has been obtained from parent/legally authorized representative
13. Written minor assent has been obtained in accordance with local laws and IRB/IEC
requirements
14. Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable (eg, Written Authorization for Use and Release of Health and Research Study Information for US sites and written Data Protection consent for EU sites)
15. Negative urine pregnancy test at the screening and day 1 visits (for females of childbearing potential, defined as females post menarche)
16. Has family commitment to comply with protocol-required procedures
17. Patients who are appropriate candidates for the protocol-specified study treatment doses
per investigator[Single Quote]s clinical judgment